The therapy, EPI-321, is intended to treat cases of facioscapulohumeral muscular dystrophy, a genetic neuromuscular disorder.

The patient died of acute liver failure; the maker of the gene therapy noted that the patient also had a recent ...

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death of a patient taking Sarepta’s Elevidys raises important safety questions.

Regenxbio is ranked Strong Buy due to low EV, strong partnerships, royalty income, bullish trends, and buyout potential.

WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment.

Shares of Sarepta Therapeutics SRPT plunged nearly 24% last week after the company reported the death of a patient following ...

Why didn’t analysts share the same fear that gripped investors about Sarepta stock? One reason is the known risk of acute liver injury associated with adeno-associated virus vector (AAV)-based gene ...

Sarepta Therapeutics (NASDAQ:SRPT – Free Report) had its target price lowered by Scotiabank from $105.00 to $80.00 in a ...

Alnylam's Amvuttra enters ATTR-CM drug race, and Expedition Therapeutics seeks to bring Chinese drugs to the U.S.

By Deena Beasley (Reuters) -Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing ...